Muscular Atrophy, SpinalPhase 3ONWARDNCT04729907
A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
A Phase 3 study testing Nusinersen in people with Muscular Atrophy, Spinal, run by Biogen at 39 sites in 16 countries.
Trial summary
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- Nusinersen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 115 participants
- Where
- 39 sitesin 16 countries
- Study dates
- 19 Apr 2021to 30 Jul 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol
Who cannot join
- Treatment with another investigational therapy or enrollment in another interventional clinical study
- Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566)
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Trial locations and contacts
39 sites in 16 countries.
39 sites in 16 countries
United States7 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
The Johns Hopkins Hospital
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-260
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Children's Medical Center
Plano, Texas, 75024
Stanford University Medical Center
Sacramento, California, 94304
Chile3 sites
Hospital Luis Calvo Mackenna
Santiago, 7500539
Clinica Las Condes
Santiago, 7550000
Clinica MEDS La Dehesa
Santiago, 7691236
China3 sites
Peking University First Hospital
Beijing, Beijing Municipality, 100034
Guangzhou Woman and Children's Medical Center
Guangzhou, Guangdong, 510623
The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Japan3 sites
Kurume University Hospital
Kurume-shi, Fukuoka, 830-0011
Hyogo Medical University Hospital
Nishinomiya-shi, Hyōgo, 663-8501
Tokyo Women's Medical University Hospital
Shinjuku-ku, Tokyo-To, 162-8666
Mexico3 sites
Antiguo Hospital Civil de Guadalajara Fray Antonio Alcalde
Guadalajara, 44280
Instituto Nacional de Pediatria
Mexico City, Mexico City, 4530
Hospital Infantil de Mexico Federico Gomez
Mexico City, Mexico City, 6720
Saudi Arabia3 sites
King Fahad Specialist Hospital
Dammam, 31444
National Guard Health Affairs: King Abdulaziz Medical City
Jeddah, 21423
King Faisal Specialist Hospital & Research Center
Riyadh, 11211
Spain3 sites
Hospital Sant Joan de Deu
Esplugues Del Llobregat, Barcelona, 8950
Hospital Universitario La Paz
Madrid, 28046
Hospital Universitari i Politecnic La Fe
Valencia, 46026
Brazil2 sites
Hospital de Clínicas de Porto Alegre
Porto Alegre, 90035-903
Hospital das Clinicas - FMUSP
São Paulo, 5403900
Germany2 sites
Universitaetsklinikum Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, 79106
Universitaetsklinikum Giessen und Marburg GmbH
Giessen, Hesse, 35392
Italy2 sites
Fondazione Serena Onlus - Centro Clinico Nemo
Milan, 20162
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, 168
Russia2 sites
Russian Children Neuromuscular Center of Veltischev
Moskva, 125412
Regional Pediatric Clinical Hospital #1
Yekaterinburg, 620149
Taiwan2 sites
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, 807
National Taiwan University Hospital
Taipei, 100
Canada1 site
London Health Sciences Centre (LHSC) - Children's Hospital
London, Ontario, N6A 5W9
Lebanon1 site
Saint George University Hospital Medical Center
Beirut, 11 00 2807
About this study
Official title: A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203.
The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are:
Read more
- How many participants have adverse events and serious adverse events during the study?
- How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment?
- How many participants have a low platelet count after treatment?
- How many participants had a change in the time it took for their heart to recharge between beats after treatment?
- How does each participant's height and other measures of growth change after treatment?
- How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment?
Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
The 232SM302 study will be done as follows:
- Participants will be screened to check if they can join the study.
- Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study.
- Each participant will receive nusinersen once every 4 months during the treatment period.
- Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
- The treatment period will last for up to 64 months (1921 days).
- There will be a follow-up safety period that lasts from 4 to 8 weeks.
- In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
Read the detailed description
The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).
The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- Nusinersen
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NusinersenDrug
Also called BIIB058, Spinraza
Administered as specified in the treatment arm
| Study group | Role | What they receive |
|---|---|---|
| BIIB058 28 mg (Prior Maintenance Dose 28 mg) | Experimental | Participants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921. Receives: Drug: Nusinersen |
| BIIB058 50/28 mg (Prior Maintenance Dose 12 mg) | Experimental | Participants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921. Receives: Drug: Nusinersen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect, or is a medically important event.
Measured: Up to Day 1921
Change from Baseline in Growth Parameters
Growth parameters will be assessed by measuring body length or height (if feasible and appropriate), ulnar length (all participants), and head circumference, chest circumference, and arm circumference (all participants 3 years of age and younger) in centimeters.
Measured: Up to Day 1921
Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters
Measured: Up to Day 1921
Number of Participants With Shifts from Baseline in Electrocardiogram (ECG)
Measured: Up to Day 1921
Number of Participants With Shifts from Baseline in Vital Signs
Measured: Up to Day 1921
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
Measured: Up to Day 1921
Change from Baseline in Prothrombin Time (PT)
Measured: Up to Day 1921
Change from Baseline in International Normalized Ratio (INR)
Measured: Up to Day 1921
Change from Baseline in Urine Total Protein
Measured: Up to Day 1921
Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age
For participants 2 years of age and younger, the Hammersmith Infant Neurological Exam (HINE) Sections 1 and 3 will be conducted. This standard examination (developed by [Dubowitz and Dubowitz 1981]) is a quantitative scorable method for assessing the neurological development of infants between 2 and 24 months of age. The examination includes assessment of cranial nerve functions, posture, movements, tone, and reflexes. The HINE Section 1 form utilized in ONWARD contains 26 items and the Section 3 form utilized contains 3 items. For HINE Section 1 items, each item is scored 0-3. For HINE Section 3 items, scoring is variable (1-4, 1-5, or 1-6). Higher scores indicate better neurological function.
Measured: Up to Day 1921
Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age
For all participants >2 years of age, standard neurological examinations, which include assessments of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes, will be conducted.
Measured: Up to Day 1921
Percentage of Participants With a Postbaseline Platelet Count Below the Lower Limit of Normal on at least 2 Consecutive Measurements
Measured: Up to Day 1921
Percentage of Participants With a Postbaseline Corrected QT Interval Using Fridericia's Formula (QTcF) of >500 millisecond (msec) and an Increase from Baseline to Any Postbaseline Timepoint in QTcF of >60 msec
Measured: Up toDay 1921
Secondary outcomes (13)
Total Number of New World Health Organization (WHO) Motor Milestones
Measured: Up to Day 1921
Number of Participants Who Used Respiratory Support, by Type
Measured: Up to Day 1921
Number of Hours Per Day of Respiratory Support
Measured: Up to Day 1921
Number of Days That Respiratory Support Is Used
Measured: Up to Day 1921
Time to Death (Overall Survival)
Measured: Up to Day 1921
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
The CHOP INTEND test is designed to evaluate the motor skills of infants with significant motor weakness. It includes 16 items (capturing neck, trunk, and proximal and distal limb strength) structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4. The total score ranges from 0-64, with higher scores depicting better response. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Measured: Up to Day 1921
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
Section 2 of the HINE is used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp (0 to 3), ability to kick in supine position (0 to 4), head control (0 to 2), rolling (0 to 3), sitting (0 to 4), crawling (0 to 4), standing (0 to 3), and walking (0 to 3). Total HINE score is the sum of points from each item and can range from 0 to 26, with higher scores depicting better level of ability. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Measured: Up to Day 1921
Percentage of HINE Section 2 Motor Milestone Responders
Section 2 of HINE is used to assess motor milestones of participants. It is composed of 8 motor milestone categories: voluntary grasp (0 to 3), ability to kick in supine position (0 to 4), head control (0 to 2), rolling (0 to 3), sitting (0 to 4), crawling (0 to 4), standing (0 to 3), and walking (0 to 3). Total HINE score is sum of points from each item and can range from 0 to 26, with higher scores depicting better level of ability. HINE section 2 motor milestone responder is participant who demonstrates at least 2-point increase in category of ability to kick or increase to maximal score on that category or 1-point increase in motor milestones category of head control, rolling, sitting, crawling, standing, or walking, and among 7 motor milestone categories (excluding voluntary grasp), participant demonstrates improvement in more categories than worsening. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Measured: Up to Day 1921
Percentage of Time Spent on Ventilation
This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Measured: Up to Day 1921
Time to Death or Permanent Ventilation
Permanent ventilation is defined as tracheostomy or ≥16 hours of ventilation/day continuously for >21 days in the absence of an acute reversible event. This outcome measure will be assessed for participants who were evaluated with this measure in study 232SM203 (NCT04089566).
Measured: Up to Day 1921
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
The HFMSE is a tool used to assess motor function in children with SMA. The original 20 item Hammersmith Functional Motor Scale (HFMS) was expanded to include 13 additional items to improve sensitivity for the higher functioning ambulant population. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all activities, with a maximum score of 66 with higher scores depicting better ability to perform activities. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with HFMSE.
Measured: Up to Day 1921
Change from Baseline in Revised Upper Limb Module (RULM) Score
The RULM is developed to assess upper limb functional abilities participants with SMA. This test consists of upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function. Participants ≥ 2 years of age (at the time of the study visit) will be evaluated with RULM.
Measured: Up to Day 1921
Change From Baseline in Plasma Levels of Neurofilament (NF)
Measured: Up to Day 1921
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2021
- Study start19 Apr 2021
- Primary completion30 Jul 2026
- Study completion30 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 115 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04729907
- Sponsor's protocol ID
- 232SM302
- Other IDs
- 2023-505637-27, 2023
- Acronym
- ONWARD
- Status last verified
- Aug 2026
- First submitted
- 11 Dec 2020
- Last updated
- 11 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
- ClinicalTrials.gov ID
- NCT04729907
- Acronym / sponsor ID
- ONWARD / 232SM302
- Conditions
- Muscular Atrophy, Spinal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- Study start
- 19 Apr 2021 (actual)
- Primary completion
- 30 Jul 2026 (actual)
- Study completion
- 30 Jul 2026 (actual)
- First posted
- 29 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study is an extension study and will enroll only those participants who have completed treatment in the parent study, 232SM203.
The main goal of the study is to learn about the long-term safety of nusinersen. The main questions researchers want to answer are:
- How many participants have adverse events and serious adverse events during the study?
- How do the results of electrocardiograms (ECGs), vital signs, and laboratory tests including blood and urine tests change after treatment?
- How many participants have a low platelet count after treatment?
- How many participants had a change in the time it took for their heart to recharge between beats after treatment?
- How does each participant's height and other measures of growth change after treatment?
- How much do the results of neurological exams that check movement, reflexes, and brain function change after treatment?
Researchers will also learn about the effect of nusinersen on mobility using various tests. They will study body movements, reflexes, balance, and coordination. They will also record if participants need help with breathing.
The 232SM302 study will be done as follows:
- Participants will be screened to check if they can join the study.
- Participants will receive their 1st dose of nusinersen in this study about 4 months after their final dose in the parent study.
- Each participant will receive nusinersen once every 4 months during the treatment period.
- Nusinersen will be given through a lumbar puncture, which involves injecting the drug into the fluid around the spinal cord in the lower back.
- The treatment period will last for up to 64 months (1921 days).
- There will be a follow-up safety period that lasts from 4 to 8 weeks.
- In total, participants will have up to 19 study visits. Participants will stay in the study for close to 6 years.
Detailed description
The primary objective of this study is to evaluate the long-term safety and tolerability of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).
The secondary objective of this study is to evaluate the long-term efficacy of nusinersen administered intrathecally at higher doses to participants with SMA who previously participated in study 232SM203 (NCT04089566).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| BIIB058 28 mg (Prior Maintenance Dose 28 mg) | Experimental | Drug: NusinersenParticipants who received maintenance dose of 28 milligrams (mg) nusinersen in study 232SM203 (NCT04089566), will receive maintenance dose of 28 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921. |
| BIIB058 50/28 mg (Prior Maintenance Dose 12 mg) | Experimental | Drug: NusinersenParticipants who received maintenance dose of 12 mg nusinersen in study 232SM203 (NCT04089566), will receive loading dose of 50 mg nusinersen, by intrathecal injection, on Day 1, followed by maintenance dose of 28 mg nusinersen, by intrathecal injection, every 4 months, up to Day 1921. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the study protocol
Key exclusion criteria (3 listed)
- Treatment with another investigational therapy or enrollment in another interventional clinical study
- Treatment with an approved therapy for SMA after the Day 302 Visit of Study 232SM203 (NCT04089566)
- NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NusinersenAlso: BIIB058, SpinrazaAdministered as specified in the treatment arm | Drug | BIIB058 28 mg (Prior Maintenance Dose 28 mg), BIIB058 50/28 mg (Prior Maintenance Dose 12 mg) |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to Day 1921
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a birth defect, or is a medically important event.
Change from Baseline in Growth Parameters
Time frame: Up to Day 1921
Growth parameters will be assessed by measuring body length or height (if feasible and appropriate), ulnar length (all participants), and head circumference, chest circumference, and arm circumference (all participants 3 years of age and younger) in centimeters.
Number of Participants With Shifts from Baseline in Clinical Laboratory Parameters
Time frame: Up to Day 1921
Number of Participants With Shifts from Baseline in Electrocardiogram (ECG)
Time frame: Up to Day 1921
Number of Participants With Shifts from Baseline in Vital Signs
Time frame: Up to Day 1921
Change from Baseline in Activated Partial Thromboplastin Time (aPTT)
Time frame: Up to Day 1921
Change from Baseline in Prothrombin Time (PT)
Time frame: Up to Day 1921
Change from Baseline in International Normalized Ratio (INR)
Time frame: Up to Day 1921
Change from Baseline in Urine Total Protein
Time frame: Up to Day 1921
Change from Baseline in Neurological Examination Outcomes for Participants ≤2 Years of Age
Time frame: Up to Day 1921
For participants 2 years of age and younger, the Hammersmith Infant Neurological Exam (HINE) Sections 1 and 3 will be conducted. This standard examination (developed by [Dubowitz and Dubowitz 1981]) is a quantitative scorable method for assessing the neurological development of infants between 2 and 24 months of age. The examination includes assessment of cranial nerve functions, posture, movements, tone, and reflexes. The HINE Section 1 form utilized in ONWARD contains 26 items and the Section 3 form utilized contains 3 items. For HINE Section 1 items, each item is scored 0-3. For HINE Section 3 items, scoring is variable (1-4, 1-5, or 1-6). Higher scores indicate better neurological function.
Number of Participants with Change from Baseline in Neurological Examination Outcomes for Participants >2 Years of Age
Time frame: Up to Day 1921
For all participants >2 years of age, standard neurological examinations, which include assessments of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes, will be conducted.
Percentage of Participants With a Postbaseline Platelet Count Below the Lower Limit of Normal on at least 2 Consecutive Measurements
Time frame: Up to Day 1921
Percentage of Participants With a Postbaseline Corrected QT Interval Using Fridericia's Formula (QTcF) of >500 millisecond (msec) and an Increase from Baseline to Any Postbaseline Timepoint in QTcF of >60 msec
Time frame: Up toDay 1921
13 secondary outcomes
Total Number of New World Health Organization (WHO) Motor Milestones
Time frame: Up to Day 1921
Number of Participants Who Used Respiratory Support, by Type
Time frame: Up to Day 1921
Number of Hours Per Day of Respiratory Support
Time frame: Up to Day 1921
Number of Days That Respiratory Support Is Used
Time frame: Up to Day 1921
Time to Death (Overall Survival)
Time frame: Up to Day 1921
Change from Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Total Score
Time frame: Up to Day 1921
Change from Baseline in Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones
Time frame: Up to Day 1921
Percentage of HINE Section 2 Motor Milestone Responders
Time frame: Up to Day 1921
Percentage of Time Spent on Ventilation
Time frame: Up to Day 1921
Time to Death or Permanent Ventilation
Time frame: Up to Day 1921
Change from Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Time frame: Up to Day 1921
Change from Baseline in Revised Upper Limb Module (RULM) Score
Time frame: Up to Day 1921
Change From Baseline in Plasma Levels of Neurofilament (NF)
Time frame: Up to Day 1921
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
- SMA EuropeLink provided by the sponsor.
- CureSMALink provided by the sponsor.
- Muscular Dystrophy AssociationLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/
Related trials for Muscular Atrophy, Spinal
RecruitingObservational study
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingObservational study
A Study of the Safety of Nusinersen (Spinraza®) and Its Effects on Motor Function in Koreans Living With SMA Treated During Their Usual Medical Care
Muscular Atrophy, Spinal
Recruiting
Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders
Cerebral Palsy, Muscular Dystrophy
Recruiting
SMAdult - Monitoring of Movement on Spinal Muscular Atrophy
Spinal Amyotrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
39 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nusinersen in people with Muscular Atrophy, Spinal, run by Biogen at 39 sites in 16 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, Chile, China, Japan, Mexico and 11 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Up to Day 1921). The study lists 13 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.